Gene Therapy Net is the web resource for patients and professionals interested in gene therapy. The objectives of Gene Therapy Net are to be the information resource for basic and clinical research in gene therapy, cell therapy, and genetic vaccines, and to serve as a network in the exchange of information and news related to above areas. In addition, Gene Therapy Net provides an overview for sponsors and researchers of the different international regulations and guidelines associated with clinical gene therapy trials.

 

Posted on: 11 April 2014, source: Bio-ITWorld
Celladon today became the first company to receive breakthrough status for a gene therapy treatment in development. The treatment, MYDICAR, is intended to reduce the risk of heart failure in patients with a deficiency of the enzyme SERCA2a. The FDA made its decision to grant breakthrough status to MYDICAR on the basis of Celladon's Phase 1 trial of 39 systolic dysfunction patients. The study reported that subjects receiving high doses of MYDICAR experienced fewer heart failure events than subjects given a placebo, by a factor of more than 80%, and that this reduction was sustained for three years after treatment. No safety issues were reported in this initial trial. Breakthrough status, which is reserved for treatments targeting life-threatening diseases that show significant improvements over the standard of care, allows closer communication with the FDA and potentially an accelerated approval process.

Posted on: 20 March 2014, source: HealthCanal
Physicians and scientists from Munich and Heidelberg have now proven the long-term effectiveness of stem cell gene therapy, based on a study of patients from the first clinical trial worldwide using gene therapy to treat Wiskott-Aldrich syndrome. Yet several years after the therapy, the researchers also observed an increased incidence of acute leukemia among the patients.

Posted on: 13 March 2014, source: International Gene Therapy Consortium
Established at the meeting of the American Society of Gene and Cell Therapy (ASCGT) in May 2013, the Consortium, which is comprised of leading scientists and researchers from more than 50 locations worldwide, has created a website where patients can interact with scientists from the organization. An online course of gene therapy was created with the collaboration of many researchers to facilitate the teaching of this discipline to students in science and medicine. Patients can also ask questions about various genetic diseases. There will also be a virtual meeting place and a forum for discussion on their diseases.

Posted on: 17 February 2014, source: University of Westminster
Researchers at the University of Westminster have developed a groundbreaking method which can be used to test a new innovative cure for hepatitis C, a liver disease caused by the hepatitis C virus (HCV). The cure is the first of its kind ever to be tested in humans and comes in the form of a drug based on gene therapy which is under development by the Australian company Benitec Biopharma. Around 150 million people worldwide are infected with hepatitis C, and more than 350,000 people die every year from hepatitis C related liver diseases. Hepatitis C is one of the leading causes of liver cirrhosis and cancer, and one of the most common and seriously infectious conditions in the world.

Posted on: 29 January 2014, source: WorldBulletin
U.S. researchers have developed tiny nanoparticle robots that can travel through a patient's blood and into tumors where they deliver a therapy that turns off an important cancer gene. The finding, reported in the journal Nature on Sunday, offers early proof that a new treatment approach called RNA interference or RNAi might work in people.

Posted on: 14 January 2014, source: BioNews
Patients in a clinical trial to treat Parkinson's disease with a form of gene therapy have showed signs of significant improvements in their motor-function, according to a report published in the Lancet. Fifteen advanced-stage Parkinson's patients (three from the UK and 12 from France) were followed up a year after being injected with low, mid and high doses of a modified virus containing genes required for brain cells to produce dopamine, as part of the phase I/II study. A lack of dopamine causes patients with Parkinson's to experience tremors and difficulty in coordinating their movement. The researchers observed that after receiving the treatment the patients' scores on movement tests improved on average by 30 percent. The patients also reported having a better quality of life. Although the patients showed some side effects, overall the treatment was found to be safe and no serious side effects were observed.